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Swedish Orphan Biovitrum AB: Sobi obtains approval from the European Commission for new dosing frequency of Orfadin®

Swedish Orphan Biovitrum International AB (publ)
( (Sobi™) has received confirmation by the
European Commission (EC) approving a reduced dosing frequency for
Orfadin® (nitisinone) from twice daily to once daily, in people with
hereditary tyrosinemia type 1 (HT-1) with a body weight >20 kg.

"Sobi has a long-term commitment to improve the lives for people
affected by hereditary tyrosinemia type 1, and the above modified
dosing regimen is a further step in this journey", says Milan
Zdravkovic, Senior Vice President, Chief Medical Officer, and Head of
Research & Development at Sobi.

The approval is based on the results of a clinical study in 18 people
with HT-1, comparing a four week once-daily and four week twice daily
dosing regimen. The study showed comparable blood-levels of
nitisinone, as well as safety and efficacy for these two regimens.
There was insufficient data (few people below 20 kg bodyweight were
enrolled in the study) to be able to compare the two regimens in
people below 20 kg.


About Orfadin®
People with hereditary tyrosinemia type 1 (HT-1) have problems
breaking down an amino acid called tyrosine. Toxic by-products are
formed and accumulate in the body, which can cause liver, renal and
neurological complications. Approximately 1,000 persons worldwide are
identified as living with HT-1 today.

Orfadin® (nitisinone) blocks the breakdown of tyrosine, thereby
reducing the amount of toxic tyrosine by-products in the body.
Patients must maintain a special diet in combination with Orfadin
treatment as tyrosine is not adequately broken down. Orfadin is a
proprietary product and is developed by and made available globally
by Sobi.

Before Orfadin became available, the survival rate in HT-1 was 29 per
cent after two years for children who developed symptoms before two
months of age. [1]
After the introduction of Orfadin, the survival rate is 93 per cent
after two years in patients with treatment initiation before two
months of age.[2]

For full European prescribing information, please visit the EMA

About Sobi™
Sobi is an international specialty healthcare company dedicated to
rare diseases. Sobi's mission is to develop and deliver innovative
therapies and services to improve the lives of patients. The product
portfolio is primarily focused on Haemophilia, Inflammation and
Genetic diseases. Sobi also markets a portfolio of specialty and rare
disease products across Europe, the Middle East, North Africa and
Russia for partner companies. Sobi is a pioneer in biotechnology with
world-class capabilities in protein biochemistry and biologics
manufacturing. In 2015, Sobi had total revenues of SEK 3.2 billion
(USD 385 M) and about 700 employees. The share (STO: SOBI) is listed
on Nasdaq Stockholm. More information is available at

For more information
please contact
Media relations Investor relations
Linda Holmström, Senior Jörgen Winroth, Vice
Communications Manager President, Head of Investor
T: + 46 708 73 40 95, + T: +1 347-224-0819, +1 212
46 8 697 31 74 -579-0506, +46 8 697 2135


van Spronsen FJ, Thomasse Y, Smit GP, et al. Hepatology.

Orfadin Product monograph 12/12/2016 (Submission Control No: 193226)

Swedish Orphan Biovitrum AB
Postal address SE-112 76 Stockholm, Sweden
Phone: 46 8 697 20 00


Författare WKR

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